Vanda Pharmaceuticals Announces Presentation at 2025 AAN Annual Meeting
Vanda Pharmaceuticals (VNDA) announced its participation at the American Academy of Neurology (AAN) Annual Meeting in San Diego from April 5-9, 2025. The company will present a poster on April 9 showcasing data for VCA-894A, a novel therapeutic customized for a specific genetic mutation in Charcot-Marie-Tooth disease type 2S (CMT2S).
The presentation will highlight how VCA-894A demonstrated significant improvements in neuromuscular function using Hesperos' Human-on-a-Chip® model with patient-derived cells. Key improvements include reduced muscle fatigue and enhanced synaptic transmission between motor neurons and skeletal muscle myotubes.
The FDA has granted VCA-894A an orphan designation, and it is expected to be administered to the specific patient for whom it was developed. The experimental platform represents advancement in precision medicine, potentially enabling development of genetically-tailored treatments for unmet medical needs.
Vanda Pharmaceuticals (VNDA) ha annunciato la sua partecipazione alla Conferenza Annuale dell'Accademia Americana di Neurologia (AAN) che si terrà a San Diego dal 5 al 9 aprile 2025. L'azienda presenterà un poster il 9 aprile che mostra i dati su VCA-894A, una nuova terapia personalizzata per una specifica mutazione genetica nella malattia di Charcot-Marie-Tooth di tipo 2S (CMT2S).
La presentazione metterà in evidenza come VCA-894A abbia dimostrato significativi miglioramenti nella funzione neuromuscolare utilizzando il modello Human-on-a-Chip® di Hesperos con cellule derivate dai pazienti. I principali miglioramenti includono una riduzione della fatica muscolare e un potenziamento della trasmissione sinaptica tra i neuroni motori e i miotubi muscolari scheletrici.
La FDA ha concesso a VCA-894A una designazione di farmaco orfano, e si prevede che venga somministrato al paziente specifico per il quale è stato sviluppato. La piattaforma sperimentale rappresenta un avanzamento nella medicina di precisione, potenzialmente consentendo lo sviluppo di trattamenti su misura geneticamente per esigenze mediche insoddisfatte.
Vanda Pharmaceuticals (VNDA) anunció su participación en la Reunión Anual de la Academia Americana de Neurología (AAN) en San Diego del 5 al 9 de abril de 2025. La compañía presentará un póster el 9 de abril mostrando datos sobre VCA-894A, una nueva terapia personalizada para una mutación genética específica en la enfermedad de Charcot-Marie-Tooth tipo 2S (CMT2S).
La presentación destacará cómo VCA-894A demostró mejoras significativas en la función neuromuscular utilizando el modelo Human-on-a-Chip® de Hesperos con células derivadas de pacientes. Las principales mejoras incluyen una reducción de la fatiga muscular y una transmisión sináptica mejorada entre las neuronas motoras y los miotubos musculares esqueléticos.
La FDA ha otorgado a VCA-894A una designación de medicamento huérfano, y se espera que se administre al paciente específico para el que fue desarrollado. La plataforma experimental representa un avance en la medicina de precisión, lo que potencialmente permite el desarrollo de tratamientos adaptados genéticamente para necesidades médicas no satisfechas.
반다 제약(VNDA)는 2025년 4월 5일부터 9일까지 샌디에이고에서 열리는 미국 신경학회(AAN) 연례 회의에 참여한다고 발표했습니다. 이 회사는 4월 9일에 VCA-894A에 대한 데이터를 보여주는 포스터를 발표할 예정이며, 이는 샤르코-마리-투스병 2형(S형 CMT)의 특정 유전적 변이에 맞춤화된 새로운 치료법입니다.
발표에서는 VCA-894A가 환자 유래 세포를 사용한 Hesperos의 Human-on-a-Chip® 모델을 통해 신경근 기능에서 상당한 개선을 보여주었다는 점을 강조할 것입니다. 주요 개선 사항으로는 근육 피로 감소와 운동 뉴런과 골격근 미오튜브 간의 시냅스 전송 향상이 포함됩니다.
FDA는 VCA-894A에 대해 고아 약물 지정을 부여했으며, 이는 개발된 특정 환자에게 투여될 것으로 예상됩니다. 이 실험적 플랫폼은 정밀 의학의 발전을 나타내며, 유전적으로 맞춤화된 치료법 개발을 가능하게 할 수 있습니다.
Vanda Pharmaceuticals (VNDA) a annoncé sa participation à la Réunion Annuelle de l'Académie Américaine de Neurologie (AAN) à San Diego du 5 au 9 avril 2025. L'entreprise présentera un poster le 9 avril mettant en avant des données sur VCA-894A, une nouvelle thérapie personnalisée pour une mutation génétique spécifique dans la maladie de Charcot-Marie-Tooth de type 2S (CMT2S).
La présentation mettra en lumière comment VCA-894A a montré des améliorations significatives de la fonction neuromusculaire en utilisant le modèle Human-on-a-Chip® de Hesperos avec des cellules dérivées de patients. Les principales améliorations comprennent une réduction de la fatigue musculaire et une transmission synaptique améliorée entre les neurones moteurs et les myotubes musculaires squelettiques.
La FDA a accordé à VCA-894A une désignation de médicament orphelin, et il est prévu qu'il soit administré au patient spécifique pour lequel il a été développé. La plateforme expérimentale représente une avancée dans la médecine de précision, permettant potentiellement le développement de traitements adaptés génétiquement pour des besoins médicaux non satisfaits.
Vanda Pharmaceuticals (VNDA) gab seine Teilnahme an der Jahrestagung der American Academy of Neurology (AAN) in San Diego vom 5. bis 9. April 2025 bekannt. Das Unternehmen wird am 9. April ein Poster präsentieren, das Daten zu VCA-894A zeigt, einer neuartigen Therapie, die auf eine spezifische genetische Mutation bei der Charcot-Marie-Tooth-Krankheit Typ 2S (CMT2S) zugeschnitten ist.
Die Präsentation wird hervorheben, wie VCA-894A signifikante Verbesserungen der neuromuskulären Funktion unter Verwendung des Human-on-a-Chip®-Modells von Hesperos mit patientenabgeleiteten Zellen demonstriert hat. Zu den wichtigsten Verbesserungen gehören reduzierte Muskelermüdung und verbesserte synaptische Übertragung zwischen motorischen Neuronen und Skelettmuskel-Miotuben.
Die FDA hat VCA-894A eine Orphan-Drug-Bezeichnung erteilt, und es wird erwartet, dass es dem spezifischen Patienten, für den es entwickelt wurde, verabreicht wird. Die experimentelle Plattform stellt einen Fortschritt in der Präzisionsmedizin dar und könnte die Entwicklung genetisch maßgeschneiderter Behandlungen für unerfüllte medizinische Bedürfnisse ermöglichen.
- FDA granted orphan drug designation for VCA-894A
- Successful demonstration of therapeutic efficacy in patient-derived cell testing
- Near-term catalyst with imminent patient treatment
- Treatment to single patient with specific genetic mutation
- Early-stage development with unproven commercial viability
The following will be presented:
April 9, 2025
Presentation Title: "Translating IGHMBP2 Variants with a CMT2S Patient-specific Organ-on-a-chip Model: Personalized Medicine ASO-based Therapeutic Rescue"
Poster Presentation Session: P-12
Poster Number: 11-017
Presenter: Dr. Sandra Paulina Smieszek, Head of Genetics
This poster describes the data around a novel breakthrough therapeutic (VCA-894A) customized to a specific genetic mutation of a patient with Charcot-Marie-Tooth disease type 2S (CMT2S). The effects of VCA-894A were confirmed in Hesperos' Human-on-a-Chip® neuromuscular junction model with patient-derived cells where VCA-894A demonstrated significant improvements in neuromuscular function, including reduced muscle fatigue and improved synaptic transmission between motor neurons and skeletal muscle myotubes. The use of a microphysiological system to evaluate the effects of a genetically tailored treatment represents a significant advancement in precision medicine and the use of human relevant methods of evaluation.
VCA-894A has been granted an orphan designation by the FDA and it is expected to soon be administered to the specific patient for whom it was developed. The experimental platform described in this poster has the potential to unlock the development of treatments that can address significantly unmet medical needs based on genetic understandings and precision medicine.
Vanda has previously published an article titled "Potential ASO-based personalized treatment for Charcot-Marie-Tooth disease type 2S." In addition to the data published in the article, this poster will present some novel unpublished data on conduction velocity.
For access to the full article, visit DOI: 10.1016/j.omtn.2025.102479.
For more information on potential ASO-based personalized treatment for CMT2S, please see Vanda's prior announcement, available here.
For more information on the AAN Annual Meeting, please refer to www.aan.com.
About Vanda Pharmaceuticals Inc.
Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients. For more on Vanda Pharmaceuticals Inc., please visit www.vandapharma.com and follow us on X @vandapharma.
Corporate Contact:
Kevin Moran
Senior Vice President, Chief Financial Officer and Treasurer
Vanda Pharmaceuticals Inc.
202-734-3400
pr@vandapharma.com
Jim Golden / Jack Kelleher / Dan Moore
Collected Strategies
VANDA-CS@collectedstrategies.com
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SOURCE Vanda Pharmaceuticals Inc.
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